FDA Approves First-in-class Evkeeza™ (evinacumab-dgnb) for Patients with Ultra-rare Inherited Form of High Cholesterol
Regeneron Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) approved EvkeezaTM (evinacumab-dgnb) as an adjunct to other low-density lipoprotein cholesterol (LDL-C) lowering therapies to treat adult and pediatric patients aged 12 years and older with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first FDA-approved treatment that binds to and blocks the function of angiopoietin-like 3 (ANGPTL3), a protein that plays a key role in lipid metabolism.
The FDA approval is based on results from the Phase 3 ELIPSE HoFH trial, published in the New England Journal of Medicine (NEJM) in August 2020. In the trial, 65 patients were randomized to receive either Evkeeza 15 mg/kg intravenously every four weeks (n=43) plus other lipid-lowering therapies, compared to lipid-lowering therapies alone (placebo, n=22). The mean baseline LDL-C level of patients in both groups was 255 mg/dL.
To read the full press release announcing the FDA decision, CLICK HERE.